Clinical trial

Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)

Name

IRB202301491

Description

Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.

Trial arms

Trial start

2024-03-01

Estimated PCD

2026-11-01

Trial end

2026-11-01

Status

Not yet recruiting

Phase

Early phase I

Treatment

Tadalafil

Patients will be given either the intervention medication or a placebo (double-blinded, randomized trial) and will be asked to take the medication every day for 6 months in conjunction with weekly exercise sessions.

Arms:

Tadalafil and Exercise Arm

Placebo

Arms:

Placebo and Exercise Arm

Exercise Training

The home-based cycling exercise training program is designed to improve muscle strength and endurance. Participants will engage in individualized exercise sessions up to four times per week, lasting up to 40 minutes each. Live video and heart rate monitoring will ensure proper exercise performance and allow for adjustments to the program throughout the study. The participants will receive the exercise equipment for use at home.

Arms:

Placebo and Exercise Arm, Tadalafil and Exercise Arm

Size

Primary endpoint

Vascular responsiveness after muscle contraction to a single dose of tadalafil.

up to 4 weeks after the completion of Visits 1 and 2 of Aim 1.

Cycling time to fatigue

Baseline and 6 month follow-up visits of Aim 2.

Eligibility criteria

Inclusion Criteria: * Diagnosis of DMD confirmed by genetic report * Minimum entry age of 6.0 years old * Ambulatory * On stable glucocorticoid regimen (for \> 3 months) Exclusion Criteria: * Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable/unwilling to perform test) * Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \<45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension * Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease) * Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis * Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism * Participation in currently approved FDA trials or other investigational clinical trials during the period of the study

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Participants are assigned to receive interventions based on their completion and results of Aim 1 (responsiveness to study drug). If participants are found to be Tadalafil-responsive after one dose, they will be included in Aim 2 of the study and will be asked to participate in the double-blinded, randomized, placebo-controlled longitudinal medication \\& exercise 6-month study program.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}

Updated at

2024-03-21

1 organization

2 products

7 indications

Organization

University of Florida

Product

Tadalafil

Indication

Duchenne muscular dystrophy

Indication